Regulators in the European Union (EU) have taken several initiatives in the past four years to increase the efficiency of pediatric regulatory processes and boost the development of medicines for children. These achievements are highlighted in the closing report of the EMA and European Commission (EC) action plan on pediatrics. Some of the key improvements brought by the paediatric action plan include:

  • Strengthened focus on unmet medical needs: over the last four years, EMA and relevant stakeholders systematically got together to better identify and raise awareness on the areas where medicines for children are particularly needed, with a goal to shift the research agenda to these areas. For example, multi-stakeholder strategy fora to discuss and agree the needs of children with cancer, and of children with inflammatory bowel disease have taken place. The learnings from these meetings involving clinicians, pediatric patients and their representatives, academia, regulators, HTAs bodies, and developers are being taken into account by EMA when discussing Paediatric Investigation Plans (PIPs) for new medicines.
  • Adapting regulatory processes to better support innovation: to facilitate the establishment of PIPs, regulatory processes have been adapted and processes overall simplified. A major outcome of this work is the launch of a pilot phase for a ‘stepwise PIP’ agreement: under this framework, it will be possible in certain cases to agree on a partial development program, conditional on the development of a full PIP once evidence becomes available over time. This will allow agreeing on PIPs for innovative medicines where crucial information needed to define certain parts of the plan is not yet available while planning the conditions and milestones for companies to return to EMA’s Paediatric Committee (PDCO) and discuss the uncertainties once more data are available. More information on this newly developed framework, launched today, is provided in the guidance for a stepwise PIP pilot.
  • Increased alignment of data requirements between decision-makers: to facilitate the compatibility of pediatric requirements between regulators, EMA has strengthened its collaboration with international partners, and notably within its pediatric cluster with the US Food and Drug Administration (FDA) and other international regulators. Work was also carried out through the European network of pediatric research at EMA (Enpr-EMA) to align international requirements for pediatric clinical trials authorization and standards.

Read more here.

References

  1. Ema. (2023, February 6). Actions to support the development of medicines for children – European Medicines Agency. European Medicines Agency. https://www.ema.europa.eu/en/news/actions-support-development-medicines-children

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